Aegerion Pharma $AEGR held its first quarter 2012 earnings call on May 3rd. Here are a few quick notes regarding their drug candidate lomitapide and its potential competitor KYNAMRO (mipomersen) from Isis Pharma $ISIS.

US NDA and EU MAA have been accepted for review

Expect FDA advisory committee meeting late 3q or early 4q 2012

Expect US approval late 2012 and EU approval first half 2013

Project profitability in 2014

Working on patient registry for homozygous familial hypercholesterolemia (HoFH), won't say how many patients are on it until closer to launch

Expect mipomersen and lomitapide to be revieweed at same panel. Topics of discussion could include trial design, liver safety, REMS

AEGR has submitted a proposed REMS to FDA in NDA filing focused around strict on-label use and monitoring of liver enzymes.

Don't see a place for PCSK9 drugs in HoFH

Now can't forecast a date for access program/reimbursement in France (had been projecting by year-end 2011)

Need to reformulate before starting pediatric trials -won't start until 2013, and probably after initial regulatory decisions. Need to get same bioavailability, stability, palatability for whatever presentation given to kids - so even if plan to open capsules and mix with food, would have to do the work. Liquid formulation has not been tried but should be technically feasible.

Market size estimate ~3000 patients in US holds up across multiple methods of calculation so far.

In these patients, neither mipomersen nor lomitapide is going to get all patient to goal LDL levels

Intellectual property: Excited about patent issued in 2011 (administration patent through 2025-2026 in US/EU). Composition of matter (COM) patent expires in 2015, Hatch-Waxman extension through mid-2020. 10 years EU market exclusivity. Patent term extension for COM in US "yet to be determined"